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A randomized, placebo-controlled, double-blind study of sapropterin to treat ADHD symptoms and executive function impairment in children and adults with sapropterin-responsive phenylketonuria
Journal article   Open access   Peer reviewed

A randomized, placebo-controlled, double-blind study of sapropterin to treat ADHD symptoms and executive function impairment in children and adults with sapropterin-responsive phenylketonuria

B. Burton, M. Grant, A. Feigenbaum, R. Singh, R. Hendren, K. Siriwardena, J. Phillips, A. Sanchez-Valle, S. Waisbren, J. Gillis, …
Molecular genetics and metabolism, v 114(3), pp 415-424
01 Mar 2015
DOI: https://doi.org/10.1016/j.ymgme.2014.11.011
PMID: 25533024
Featured in Collection :   UN Sustainable Development Goals @ Drexel
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https://doi.org/10.1016/j.ymgme.2014.11.011View
Published, Version of Record (VoR)CC BY-NC-ND V4.0,  Open

Abstract

Endocrinology & Metabolism Genetics & Heredity Life Sciences & Biomedicine Medicine, Research & Experimental Research & Experimental Medicine Science & Technology
Symptoms of attention deficit-hyperactivity disorder (ADHD), particularly inattention, and impairments in executive functioning have been reported in early and continuously treated children, adolescents, and adults with phenylketonuria (PKU). In addition, higher blood phenylalanine (Phe) levels have been correlated with the presence of ADHD symptoms and executive functioning impairment. The placebo-controlled PKU ASCEND study evaluated the effects of sapropterin therapy on PKU-associated symptoms of ADHD and executive and global functioning in individuals who had a therapeutic blood Phe response to sapropterin therapy. The presence of ADHD inattentive symptoms and executive functioning deficits was confirmed in this large cohort of 206 children and adults with PKU, of whom 118 responded to sapropterin therapy. In the 38 individuals with sapropterin-responsive PKU and ADHD symptoms at baseline, sapropterin therapy resulted in a significant improvement in ADHD inattentive symptoms in the first 4 weeks of treatment, and improvements were maintained throughout the 26 weeks of treatment. Sapropterin was well-tolerated with a favorable safety profile. The improvements in ADHD inattentive symptoms and aspects of executive functioning in response to sapropterin therapy noted in a large cohort of individuals with PKU indicate that these symptoms are potentially reversible when blood Phe levels are reduced. (C) 2014 BioMarin Pharmaceutical Inc. Published by Elsevier Inc.

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Industry collaboration
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Web of Science research areas
Endocrinology & Metabolism
Genetics & Heredity
Medicine, Research & Experimental
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